The Past, Present, and Future of Gene Therapy

Abstract

Since 2010, gene therapy has rapidly gained interest as a possible method to cure previously untouchable and incurable diseases. The idea of tackling the disease at its genetic core to prevent the malignance from manifesting in the first place seemed unrealistic at first, but decades of research have started to bear fruit and these untouchable diseases suddenly seem mortal. CRISPR-Cas9, Chimeric Antigen Receptor (CAR) T-cell therapy, and adeno-associated virus (AAV) therapy utilize different gene engineering techniques to nullify previously incurable diseases. This paper serves as a comprehensive review paper which analyzes the mechanism, advantages, and disadvantages of these three gene therapy techniques. This paper states the genetic scissor mechanism of the CRISPR-Cas9 complex, and its difference from its predecessors, the artificial tinkering of the CAR t-cell therapy method and its subsequent utilization of the host’s immune system, and finally the transduction potential of the AAV gene therapy. Finally, this paper states the current status and clinical application of the three gene therapy techniques, including their medication terminology and target diseases, and helps elucidate the future and potential of these three gene therapy techniques.